Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD
a study on Macular Degeneration Copy Number Variation
Summary
- Eligibility
- for people ages 50-89 (full criteria)
- Location
- at UC Irvine UCSF
- Dates
- study startedcompletion around
- Principal Investigator
- by Robert Bhisitkul (ucsf)
Description
Summary
RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. RGX-314 is being developed as a potential one-time treatment for wet AMD.
Official Title
A Randomized, Partially Masked, Controlled, Phase 2b/3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD (ATMOSPHERE)
Details
This randomized, partially masked, controlled, Phase 2b/3 clinical study will evaluate the efficacy and safety of RGX-314 gene therapy in participants with nAMD. The study will evaluate 2 dose levels of RGX-314 relative to an active comparator. The primary endpoint of this study is mean change in best-corrected visual acuity (BCVA) of RGX-314 relative to ranibizumab. Approximately 300 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms.
Keywords
AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, Wet AMD, CNV, Age-Related Macular Degeneration, Neovascular Age-Related Macular Degeneration, Macular Degeneration, Wet Macular Degeneration, Choroidal Neovascularization, Retinal Degeneration, Retinal Diseases, Eye Diseases, Ranibizumab, Angiogenesis Inhibitors, Angiogenesis Modulating Agents, Growth Substances, Physiological Effects of Drugs, Growth Inhibitors, Antineoplastic Agents, Gene therapy, Anti-vascular endothelial grown factory therapy, Anti-VEGF therapy, RGX-314, Ranibizumab (LUCENTIS®)
Eligibility
You can join if…
Open to people ages 50-89
- Age ≥ 50 years and ≤ 89 years
- An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
- Diagnosis of subfoveal CNV secondary to AMD in the study eye previously treated with anti-VEGF
- Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
- Willing and able to provide written, signed informed consent for this study
- Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry
You CAN'T join if...
- CNV or macular edema in the study eye secondary to any causes other than AMD
- Subfoveal fibrosis or atrophy in the study eye
- Any condition in the investigator's opinion that could limit VA improvement in the study eye
- Active or history of retinal detachment in the study eye
- Uncontrolled glaucoma in the study eye
- History of intraocular surgery in the study eye within 12 weeks prior to Screening Visit 1
- History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1.
- Prior treatment with gene therapy.
- Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months.
Locations
- University of California Irvine - School of Medicine - The Gavin Herbert Eye Institute
accepting new patients
Irvine California 92697 United States - UCSF Medical Center - Retina and Vitreous Clinic
accepting new patients
San Francisco California 94143 United States - West Coast Retina Medical Group - San Francisco Office
accepting new patients
San Francisco California 94109 United States - Retina Vitreous Assoc Med Grp
accepting new patients
Beverly Hills California 90211 United States - Retina-Vitreous Associates Medical Group
accepting new patients
Beverly Hills California 90211 United States - The Retina Partners - Encino
accepting new patients
Encino California 91436 United States - Orange County Retina Medical Group
accepting new patients
Santa Ana California 92705 United States - Retina Consultants San Diego
accepting new patients
Poway California 92064 United States - Doheny Eye Institute - Doheny Eye Center - Arcadia
accepting new patients
Los Angeles California 90033 United States - Retina Consultants
accepting new patients
Sacramento California 95819 United States
Lead Scientist at University of California Health
- Robert Bhisitkul (ucsf)
Professor, Ophthalmology, School of Medicine. Authored (or co-authored) 62 research publications
Details
- Status
- accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- AbbVie
- ID
- NCT04704921
- Phase
- Phase 2/3 research study
- Study Type
- Interventional
- Participants
- Expecting 300 study participants
- Last Updated